Treating ATTR-CM Early: A Shift From Reactive to Strategic Care

Announcer:
This is Heart Matters on ReachMD. On this episode, Dr. Quan Bui will discuss quality of life considerations in patients with transthyretin amyloid cardiomyopathy, or ATTR-CM. Dr. Bui is a cardiologist and an Assistant Professor of Medicine at UC San Diego Health. Let’s hear from him now.

Dr. Bui:
I think quality of life depends on what stage of amyloid that you’re in. Obviously, if you’re very early on in the disease, your quality of life could be quite good. You’re relatively asymptomatic from disease. But obviously, if things progress over time—and usually, that’s what happens in transthyretin cardiac amyloidosis—you can develop end-stage heart failure and really have the classic symptoms of shortness of breath with exertion, fluid retention, etc.

Historically, in the past we didn’t have any treatments for amyloidosis, and now, over the last five years or so, we’ve had multiple treatments being developed and into clinical practice. And I’ve been quite surprised about the degree of symptom improvement in some of my patients with amyloidosis once they start some of these therapies—people even with advanced disease where I think their heart failure might be a little bit difficult to manage, they get on these amyloid specific treatments, and their symptoms of heart failure have drastically improved. They’re able to feel better from a fluid standpoint. They’re able to walk longer distances. They have improvements in their fatigue.

And so I think that these medications have been huge, and I think that the earlier you start these medications, the more effective they are, and that’s why it’s so important to diagnose these patients early. We’re moving in cardiology from a reactive approach to a more proactive one in terms of treatment. So there’s this clinical trial called ACT-EARLY where they’re actually putting patients on stabilizer therapy to see if you can prevent development of amyloidosis. And this is hopefully a new era of precision medicine where we’re trying to prevent disease rather than just treating patients once they develop symptoms or have amyloidosis.

I think quality of life is really difficult to assess in a systemic disease like amyloidosis where multiple organ systems are involved, but what I found most effective is really taking the time to listen to patients. This will help you understand what the extracardiac symptoms of this disease are and allow for appropriate referral to other specialists who can help you in managing those.

Announcer:
That was Dr. Quan Bui discussing quality of life in patients with ATTR-CM. To access this and other episodes in our series, visit Heart Matters on ReachMD.com, where you can Be Part of the Knowledge. Thanks for listening!